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Studies show promising results for new cystic fibrosis drug

7 November 2017

The results of two global clinical research trials in cystic fibrosis (CF) medicine – led by researchers at Royal Brompton Hospital – were presented at the North American CF conference in Indianapolis last week.

The phase III studies, published in the New England Journal of Medicine on Friday (3 November), revealed the impact of using the compound tezacaftor in combination with ivacaftor for people with various combinations of the CF gene.

The EVOLVE study involved patients with two copies of the ‘F508 del’ mutation, and was led by Professor Stuart Elborn, consultant in respiratory medicine and the Trust’s lead for CF.

It found that the tezacaftor/ivacaftor combination produced similar benefits to the ivacaftor/lumacaftor combination known as Orkambi, of around four per cent lung function improvement, but resulted in patients experiencing fewer side effects.

Professor Elborn said: “The combination of tezacaftor and ivacaftor used in these trials is an important step forward in the treatment of CF, and could set the scene for trials of a three-drug combination in the future. The Cystic Fibrosis Trust’s Clinical Trials Accelerator Platform will be a valuable resource in drug development like this, and we’re delighted to be participating in it."

The EXPAND study, led by Professor Jane Davies, honorary consultant in paediatric respiratory medicine, found an average improvement in lung function of 6.8 per cent in those with one ‘F508del’ and one ‘residual function’ mutation, when compared with the placebo.

Professor Davies said: “We are pleased to have played a part in the EXPAND trial and are very excited by the results. Despite having some CFTR protein function, people with this type of gene mutation can experience very significant disease. In this first trial to focus on them as a group, the demonstration that tezacaftor/ivacaftor leads to clinically meaningful improvements in lung function is a major step forward.”

Dr Keith Brownlee, Director of Impact at the Cystic Fibrosis Trust, said: “This is a very exciting time for people with CF and precision medicine. Phase III trials like these are critical, and they cannot happen unless people take part.

“That is why the Trust set up the Clinical Trials Accelerator Platform, to support access to CF clinical trials – and ultimately to help us stop the clock on cystic fibrosis.”